Small Molecule and Biotechnology-Based Therapeutics for Neurology-Focused Companies: 2021’s IPOs Part V

There has been substantial interest in the development of screening methods capable of surveying a wider sample of chemical space and larger numbers of compounds. Over the past two decades, DEL technology has evolved into a broadly utilized and validated platform in drug discovery, yielding diverse hits for a wide range of biologically relevant targets. Read the full review for a historical and technical overview of DEL technology, including how libraries are prepared and screened, a comparison to other HTS methods, and examples of successful applications in drug discovery.

The discovery that individuals with null mutations in the NaV1.7 exhibited pain insensitivity sparked interest in targeting NaV1.7 to potentially treat pain. Despite the potential of selectively inhibiting sodium channels like NaV1.7, NaV1.8, and NaV1.9 for pain management, developing selective inhibitors suitable for clinical use has proven challenging. This article complements our coverage of VX-548, NaV1.8 as a critical target in pain management, and our NaV1.8 compound roundup and provides a reminder of noteworthy preclinical and clinical NaV1.7 small molecule inhibitors as of April 2024.

Peptidic GLP-1R agonists have received significant media coverage over the past year for their astounding efficacy in several indications. While most of the recent fanfare has been related to their role in weight loss, GLP-1R agonists have been a mainstay of the type 2 diabetes treatment landscape for the past 2 decades. GLP-1R agonists have also recently shown efficacy in reducing heart disease risk and treating certain chronic kidney diseases. Here’s a recap of the March 14th, 2024, Flash Talk, from Oliver Philps.

DILI is a leading cause of acute liver failure, accounting for half of these cases and often resulting in drug withdrawals. Understanding and managing DILI risk as a medicinal chemist involves exploring the intricate interplay among properties and structural features. In this article, we provide a medicinal chemist's perspective on the current understanding of DILI mechanisms, highlight cutting-edge assay developments for a holistic assessment, and discuss strategies for predicting DILI risks.

This article compiles 20+ recent small molecules of general interest in the news in April 2023, with structures where they are available. Capacity Bio’s MAS Receptor Agonists with $35M Raised for FIH Clinical Trials MAS modulator from WO2022165189 example 107 New start-up Capacity Bio backed by RA Capital, Insight Partners, and Remiges [...]